Brooke Krivickas, Erica Scirocco, Elisa Giacomelli, Saloni Sharma, Molly Benson, Mackenzie Keegan, Jennifer Kulesa-Kelley, Lori B Chibnik, Gabriella Casagrande, Lindsay Heyd, Marianne Chase, Kristin Drake, Silpa Mohapatra, Jennifer Linn Hagar, Meredith Gibbons Hasenoehrl, Derek Dagostino, Alexander V Sherman, Alex Leite, Hong Yu, Jesse Rosenthal, Timothy Miller, Alexandra McCaffrey, Kelly Gwathmey, Eduardo Locatelli, Elham Bayat, Daragh Heitzman, Eufrosina Young, Namita A Goyal, Jackie Whitesell, Kevin Felice, Hristelina Ilieva, Andrea Swenson, David Walk, Gustavo Alameda, Laura Foster, Courtney E McIlduff, Alison Walsh, Lindsay Zilliox, Senda Ajroud-Driss, Cynthia Bodkin, Jonathan Katz, Shafeeq Ladha, Michael Rivner, Laura Rosow, Paul Twydell, Warren Wasiewski, Suma Babu, James D Berry, Sabrina Paganoni
{"title":"肌萎缩性侧索硬化症患者海藻糖获取研究的多中心扩展准入协议。","authors":"Brooke Krivickas, Erica Scirocco, Elisa Giacomelli, Saloni Sharma, Molly Benson, Mackenzie Keegan, Jennifer Kulesa-Kelley, Lori B Chibnik, Gabriella Casagrande, Lindsay Heyd, Marianne Chase, Kristin Drake, Silpa Mohapatra, Jennifer Linn Hagar, Meredith Gibbons Hasenoehrl, Derek Dagostino, Alexander V Sherman, Alex Leite, Hong Yu, Jesse Rosenthal, Timothy Miller, Alexandra McCaffrey, Kelly Gwathmey, Eduardo Locatelli, Elham Bayat, Daragh Heitzman, Eufrosina Young, Namita A Goyal, Jackie Whitesell, Kevin Felice, Hristelina Ilieva, Andrea Swenson, David Walk, Gustavo Alameda, Laura Foster, Courtney E McIlduff, Alison Walsh, Lindsay Zilliox, Senda Ajroud-Driss, Cynthia Bodkin, Jonathan Katz, Shafeeq Ladha, Michael Rivner, Laura Rosow, Paul Twydell, Warren Wasiewski, Suma Babu, James D Berry, Sabrina Paganoni","doi":"10.1002/mus.70011","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction/aims: </strong>Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)-funded EAP for amyotrophic lateral sclerosis (ALS).</p><p><strong>Methods: </strong>Participants received trehalose, a drug studied in a parallel RCT, for up to 24 weeks; clinical and biomarker data were collected throughout the study.</p><p><strong>Results: </strong>Seventy participants were enrolled at 20 study centers across the United States. Treatment with trehalose did not affect the levels of neurofilament light chain [estimated flat slope per month was -0.005, SE = 0.0078; 95% CI (-0.021, 0.011)] or disease progression [estimated least square mean change of the ALS Functional Rating Scale-Revised total score and slow vital capacity (percent predicted) from baseline to Week 24 were -5.6 (0.67); 95% CI (-7.0, -4.3) and -4.53 (4.308); 95% CI (-13.55, 4.48)], respectively. No unexpected treatment-related risks were identified. Serious adverse events were deemed not related to trehalose (20 occurrences in 13 [18.6%] participants with eight deaths).</p><p><strong>Discussion: </strong>This EAP establishes a framework for implementing multi-center EAPs that complement data collected from RCTs. Additional NIH-funded EAPs are currently underway. Data and additional serum samples collected in this study are available to the research community for further study.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov: NCT05597436.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"1108-1116"},"PeriodicalIF":3.1000,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12428506/pdf/","citationCount":"0","resultStr":"{\"title\":\"Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis.\",\"authors\":\"Brooke Krivickas, Erica Scirocco, Elisa Giacomelli, Saloni Sharma, Molly Benson, Mackenzie Keegan, Jennifer Kulesa-Kelley, Lori B Chibnik, Gabriella Casagrande, Lindsay Heyd, Marianne Chase, Kristin Drake, Silpa Mohapatra, Jennifer Linn Hagar, Meredith Gibbons Hasenoehrl, Derek Dagostino, Alexander V Sherman, Alex Leite, Hong Yu, Jesse Rosenthal, Timothy Miller, Alexandra McCaffrey, Kelly Gwathmey, Eduardo Locatelli, Elham Bayat, Daragh Heitzman, Eufrosina Young, Namita A Goyal, Jackie Whitesell, Kevin Felice, Hristelina Ilieva, Andrea Swenson, David Walk, Gustavo Alameda, Laura Foster, Courtney E McIlduff, Alison Walsh, Lindsay Zilliox, Senda Ajroud-Driss, Cynthia Bodkin, Jonathan Katz, Shafeeq Ladha, Michael Rivner, Laura Rosow, Paul Twydell, Warren Wasiewski, Suma Babu, James D Berry, Sabrina Paganoni\",\"doi\":\"10.1002/mus.70011\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction/aims: </strong>Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)-funded EAP for amyotrophic lateral sclerosis (ALS).</p><p><strong>Methods: </strong>Participants received trehalose, a drug studied in a parallel RCT, for up to 24 weeks; clinical and biomarker data were collected throughout the study.</p><p><strong>Results: </strong>Seventy participants were enrolled at 20 study centers across the United States. Treatment with trehalose did not affect the levels of neurofilament light chain [estimated flat slope per month was -0.005, SE = 0.0078; 95% CI (-0.021, 0.011)] or disease progression [estimated least square mean change of the ALS Functional Rating Scale-Revised total score and slow vital capacity (percent predicted) from baseline to Week 24 were -5.6 (0.67); 95% CI (-7.0, -4.3) and -4.53 (4.308); 95% CI (-13.55, 4.48)], respectively. No unexpected treatment-related risks were identified. Serious adverse events were deemed not related to trehalose (20 occurrences in 13 [18.6%] participants with eight deaths).</p><p><strong>Discussion: </strong>This EAP establishes a framework for implementing multi-center EAPs that complement data collected from RCTs. Additional NIH-funded EAPs are currently underway. Data and additional serum samples collected in this study are available to the research community for further study.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov: NCT05597436.</p>\",\"PeriodicalId\":18968,\"journal\":{\"name\":\"Muscle & Nerve\",\"volume\":\" \",\"pages\":\"1108-1116\"},\"PeriodicalIF\":3.1000,\"publicationDate\":\"2025-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12428506/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Muscle & Nerve\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1002/mus.70011\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/8/26 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q2\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Muscle & Nerve","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/mus.70011","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/8/26 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis.
Introduction/aims: Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)-funded EAP for amyotrophic lateral sclerosis (ALS).
Methods: Participants received trehalose, a drug studied in a parallel RCT, for up to 24 weeks; clinical and biomarker data were collected throughout the study.
Results: Seventy participants were enrolled at 20 study centers across the United States. Treatment with trehalose did not affect the levels of neurofilament light chain [estimated flat slope per month was -0.005, SE = 0.0078; 95% CI (-0.021, 0.011)] or disease progression [estimated least square mean change of the ALS Functional Rating Scale-Revised total score and slow vital capacity (percent predicted) from baseline to Week 24 were -5.6 (0.67); 95% CI (-7.0, -4.3) and -4.53 (4.308); 95% CI (-13.55, 4.48)], respectively. No unexpected treatment-related risks were identified. Serious adverse events were deemed not related to trehalose (20 occurrences in 13 [18.6%] participants with eight deaths).
Discussion: This EAP establishes a framework for implementing multi-center EAPs that complement data collected from RCTs. Additional NIH-funded EAPs are currently underway. Data and additional serum samples collected in this study are available to the research community for further study.
期刊介绍：
Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.
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